Identification of genetic modifiers of Huntington's disease somatic CAG repeat instability by in vivo CRISPR-Cas9 genome editing
Ricardo Mouro Pinto, Ryan Murtha, António Azevedo, Cameron J. Douglas, Marina V. Kovalenko, Jessica Ulloa, Steven Crescenti, Zoe N. Burch, Esaria Oliver, Maheswaran Kesavan, Shota Shibata, Antonia G. Vitalo, Eduarda Mota‐Silva, Marion J. Riggs, Kevin Correia, , Vanessa Chantal Wheeler
The paper identifies genetic modifiers of Huntington's disease somatic CAG repeat instability using CRISPR-Cas9 in HD knock-in mice, revealing new therapeutic targets.
Nature Genetics · Jan 22, 2025Peer-reviewedGenetics